-
2
-
-
0025826050
-
Lysosomal disease
-
Neufeld E.F. Lysosomal disease. Ann Rev Biochem 60 (1991) 257-280
-
(1991)
Ann Rev Biochem
, vol.60
, pp. 257-280
-
-
Neufeld, E.F.1
-
3
-
-
0027948540
-
Enzyme replacement treatment in type 1 and type 3 Gaucher's disease
-
Bembi B., Zanatta M., Carrozzi M., Baralle F., Gornati R., Berra B., et al. Enzyme replacement treatment in type 1 and type 3 Gaucher's disease. Lancet 344 (1994) 1679-1682
-
(1994)
Lancet
, vol.344
, pp. 1679-1682
-
-
Bembi, B.1
Zanatta, M.2
Carrozzi, M.3
Baralle, F.4
Gornati, R.5
Berra, B.6
-
4
-
-
0023196876
-
Prevention of deterioration in metachromatic leukodystrophy by bone marrow transplantation
-
Krivit W., Lipton M.E., Lockman L.A., Tsai M., Dyck P.J., Smith S., et al. Prevention of deterioration in metachromatic leukodystrophy by bone marrow transplantation. Am J Med Sci 294 (1987) 80-85
-
(1987)
Am J Med Sci
, vol.294
, pp. 80-85
-
-
Krivit, W.1
Lipton, M.E.2
Lockman, L.A.3
Tsai, M.4
Dyck, P.J.5
Smith, S.6
-
5
-
-
0025022733
-
Treatment of late infantile metachromatic leukodystrophy by bone marrow transplantation
-
Krivit W., Shapiro E., Kennedy W., Lipton M., Lockman L., Smith S., et al. Treatment of late infantile metachromatic leukodystrophy by bone marrow transplantation. N Engl J Med 322 (1990) 28-32
-
(1990)
N Engl J Med
, vol.322
, pp. 28-32
-
-
Krivit, W.1
Shapiro, E.2
Kennedy, W.3
Lipton, M.4
Lockman, L.5
Smith, S.6
-
6
-
-
10544235699
-
Phenotype of arylsulfatase A-deficient mice: relationship to human metachromatic leukodystrophy
-
Hess B., Saftig P., Hartmann D., Coenen R., Lullmann-Rauch R., Goebel H.H., et al. Phenotype of arylsulfatase A-deficient mice: relationship to human metachromatic leukodystrophy. Proc Natl Acad Sci USA 93 (1996) 14821-14826
-
(1996)
Proc Natl Acad Sci USA
, vol.93
, pp. 14821-14826
-
-
Hess, B.1
Saftig, P.2
Hartmann, D.3
Coenen, R.4
Lullmann-Rauch, R.5
Goebel, H.H.6
-
7
-
-
11144354757
-
Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells
-
Biffi A., De Palma M., Quattrini A., Del Carro U., Amadio S., Visigalli I., et al. Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. J Clin Invest 113 (2004) 1118-1129
-
(2004)
J Clin Invest
, vol.113
, pp. 1118-1129
-
-
Biffi, A.1
De Palma, M.2
Quattrini, A.3
Del Carro, U.4
Amadio, S.5
Visigalli, I.6
-
8
-
-
20944444685
-
Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy
-
Matzner U., Herbst E., Hedayati K.K., Lullmann-Rauch R., Wessig C., Schroder S., et al. Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy. Hum Mol Genet 14 (2005) 1139-1152
-
(2005)
Hum Mol Genet
, vol.14
, pp. 1139-1152
-
-
Matzner, U.1
Herbst, E.2
Hedayati, K.K.3
Lullmann-Rauch, R.4
Wessig, C.5
Schroder, S.6
-
9
-
-
0035049241
-
The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher's disease
-
Altarescu G., Hill S., Wiggs E., Jeffries N., Kreps C., Parker C.C., et al. The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher's disease. J Pediatr 138 (2001) 539-547
-
(2001)
J Pediatr
, vol.138
, pp. 539-547
-
-
Altarescu, G.1
Hill, S.2
Wiggs, E.3
Jeffries, N.4
Kreps, C.5
Parker, C.C.6
-
10
-
-
17044440923
-
Extended interval between enzyme therapy infusions for adult patients with Gaucher's disease type 1
-
Perez-Calvo J., Giraldo P., Pastores G.M., Fernandez-Galan M., Martin-Nunez G., and Pocovi M. Extended interval between enzyme therapy infusions for adult patients with Gaucher's disease type 1. J Postgrad Med 49 (2003) 127-131
-
(2003)
J Postgrad Med
, vol.49
, pp. 127-131
-
-
Perez-Calvo, J.1
Giraldo, P.2
Pastores, G.M.3
Fernandez-Galan, M.4
Martin-Nunez, G.5
Pocovi, M.6
-
11
-
-
0035718871
-
Enzyme replacement therapy in feline mucopolysaccharidosis I
-
Kakkis E.D., Schuchman E., He X., Wan Q., Kania S., Wiemelt S., et al. Enzyme replacement therapy in feline mucopolysaccharidosis I. Mol Genet Metab 72 (2001) 199-208
-
(2001)
Mol Genet Metab
, vol.72
, pp. 199-208
-
-
Kakkis, E.D.1
Schuchman, E.2
He, X.3
Wan, Q.4
Kania, S.5
Wiemelt, S.6
-
12
-
-
0035000479
-
Enzyme replacement therapy in mucopolysaccharidosis type I: progress and emerging difficulties
-
Wraith J.E. Enzyme replacement therapy in mucopolysaccharidosis type I: progress and emerging difficulties. J Inherit Metab Dis 24 (2001) 245-250
-
(2001)
J Inherit Metab Dis
, vol.24
, pp. 245-250
-
-
Wraith, J.E.1
-
13
-
-
0035099437
-
In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice
-
Consiglio A., Quattrini A., Martino S., Bensadoun J.C., Dolcetta D., Trojani A., et al. In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice. Nat Med 7 (2001) 310-316
-
(2001)
Nat Med
, vol.7
, pp. 310-316
-
-
Consiglio, A.1
Quattrini, A.2
Martino, S.3
Bensadoun, J.C.4
Dolcetta, D.5
Trojani, A.6
-
14
-
-
20244383300
-
AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease
-
Passini M.A., Macauley S.L., Huff M.R., Taksir T.V., Bu J., Wu I.H., et al. AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease. Mol Ther 11 (2005) 754-762
-
(2005)
Mol Ther
, vol.11
, pp. 754-762
-
-
Passini, M.A.1
Macauley, S.L.2
Huff, M.R.3
Taksir, T.V.4
Bu, J.5
Wu, I.H.6
-
15
-
-
0035203152
-
Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector
-
Passini M.A., and Wolfe J.H. Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector. J Virol 75 (2001) 12382-12392
-
(2001)
J Virol
, vol.75
, pp. 12382-12392
-
-
Passini, M.A.1
Wolfe, J.H.2
-
16
-
-
0032878803
-
Evaluation of reaction of primate brain to grafted PC12 cells
-
Yoshida H., Date I., Shingo T., Fujiwara K., Miyoshi Y., Furuta T., et al. Evaluation of reaction of primate brain to grafted PC12 cells. Cell Transplant 8 (1999) 427-430
-
(1999)
Cell Transplant
, vol.8
, pp. 427-430
-
-
Yoshida, H.1
Date, I.2
Shingo, T.3
Fujiwara, K.4
Miyoshi, Y.5
Furuta, T.6
-
17
-
-
0031239671
-
Long-term enhanced chromaffin cell survival and behavioral recovery in hemiparkinsonian rats with co-grafted polymer-encapsulated human NGF-secreting cells
-
Date I., Shingo T., Ohmoto T., and Emerich D.F. Long-term enhanced chromaffin cell survival and behavioral recovery in hemiparkinsonian rats with co-grafted polymer-encapsulated human NGF-secreting cells. Exp Neurol 147 (1997) 10-17
-
(1997)
Exp Neurol
, vol.147
, pp. 10-17
-
-
Date, I.1
Shingo, T.2
Ohmoto, T.3
Emerich, D.F.4
-
18
-
-
0032077263
-
Dose-dependent doxycycline-mediated adrenocorticotropic hormone secretion from encapsulated Tet-on proopiomelanocortin Neuro2A cells in the subarachnoid space
-
Saitoh Y., Eguchi Y., Hagihara Y., Arita N., Watahiki M., Tsujimoto Y., et al. Dose-dependent doxycycline-mediated adrenocorticotropic hormone secretion from encapsulated Tet-on proopiomelanocortin Neuro2A cells in the subarachnoid space. Hum Gene Ther 9 (1998) 997-1002
-
(1998)
Hum Gene Ther
, vol.9
, pp. 997-1002
-
-
Saitoh, Y.1
Eguchi, Y.2
Hagihara, Y.3
Arita, N.4
Watahiki, M.5
Tsujimoto, Y.6
-
19
-
-
0030906578
-
Protective effect of encapsulated cells producing neurotrophic factor CNTF in a monkey model of Huntington's disease
-
Emerich D.F., Winn S.R., Hantraye P.M., Peschanski M., Chen E.Y., Chu Y., et al. Protective effect of encapsulated cells producing neurotrophic factor CNTF in a monkey model of Huntington's disease. Nature 386 (1997) 395-399
-
(1997)
Nature
, vol.386
, pp. 395-399
-
-
Emerich, D.F.1
Winn, S.R.2
Hantraye, P.M.3
Peschanski, M.4
Chen, E.Y.5
Chu, Y.6
-
20
-
-
15844381714
-
Intrathecal delivery of CNTF using encapsulated genetically modified xenogeneic cells in amyotrophic lateral sclerosis patients
-
Aebischer P., Schluep M., Deglon N., Joseph J.M., Hirt L., Heyd B., et al. Intrathecal delivery of CNTF using encapsulated genetically modified xenogeneic cells in amyotrophic lateral sclerosis patients. Nat Med 2 (1996) 696-699
-
(1996)
Nat Med
, vol.2
, pp. 696-699
-
-
Aebischer, P.1
Schluep, M.2
Deglon, N.3
Joseph, J.M.4
Hirt, L.5
Heyd, B.6
-
21
-
-
0034633677
-
Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells
-
Ross C.J., Bastedo L., Maier S.A., Sands M.S., and Chang P.L. Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells. Hum Gene Ther 11 (2000) 2117-2127
-
(2000)
Hum Gene Ther
, vol.11
, pp. 2117-2127
-
-
Ross, C.J.1
Bastedo, L.2
Maier, S.A.3
Sands, M.S.4
Chang, P.L.5
-
22
-
-
27944473627
-
Reduction of GAG storage in MPS II mouse model following implantation of encapsulated recombinant myoblasts
-
Friso A., Tomanin R., Alba S., Gasparotto N., Puicher E.P., Fusco M., et al. Reduction of GAG storage in MPS II mouse model following implantation of encapsulated recombinant myoblasts. J Gene Med 7 (2005) 1482-1491
-
(2005)
J Gene Med
, vol.7
, pp. 1482-1491
-
-
Friso, A.1
Tomanin, R.2
Alba, S.3
Gasparotto, N.4
Puicher, E.P.5
Fusco, M.6
-
23
-
-
0024541384
-
Cloning and expression of human arylsulfatase A
-
Stein C., Gieselmann V., Kreysing J., Schmidt B., Pohlmann R., Waheed A., et al. Cloning and expression of human arylsulfatase A. J Biol Chem 264 (1989) 1252-1259
-
(1989)
J Biol Chem
, vol.264
, pp. 1252-1259
-
-
Stein, C.1
Gieselmann, V.2
Kreysing, J.3
Schmidt, B.4
Pohlmann, R.5
Waheed, A.6
-
24
-
-
0024460181
-
Improved retroviral vectors for gene transfer and expression
-
Miller A.D., and Rosman G.J. Improved retroviral vectors for gene transfer and expression. Biotechniques 7 (1989) 980-990
-
(1989)
Biotechniques
, vol.7
, pp. 980-990
-
-
Miller, A.D.1
Rosman, G.J.2
-
25
-
-
0028795530
-
Transfer of the HSV-tk gene into donor peripheral blood lymphocytes for in vivo modulation of donor anti-tumor immunity after allogeneic bone marrow transplantation
-
Bordignon C., Bonini C., Verzeletti S., Nobili N., Maggioni D., Traversari C., et al. Transfer of the HSV-tk gene into donor peripheral blood lymphocytes for in vivo modulation of donor anti-tumor immunity after allogeneic bone marrow transplantation. Hum Gene Ther 6 (1995) 813-819
-
(1995)
Hum Gene Ther
, vol.6
, pp. 813-819
-
-
Bordignon, C.1
Bonini, C.2
Verzeletti, S.3
Nobili, N.4
Maggioni, D.5
Traversari, C.6
-
26
-
-
49749198315
-
The assay of arylsulfatase A and B in human urine
-
Baum J., Dodgson K.S., and Spencer B. The assay of arylsulfatase A and B in human urine. Clin Chim Acta 4 (1959) 453-455
-
(1959)
Clin Chim Acta
, vol.4
, pp. 453-455
-
-
Baum, J.1
Dodgson, K.S.2
Spencer, B.3
-
27
-
-
0017184389
-
A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding
-
Bradford M.M. A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding. Anal Biochem 72 (1976) 248-254
-
(1976)
Anal Biochem
, vol.72
, pp. 248-254
-
-
Bradford, M.M.1
-
28
-
-
0020059002
-
Beta-glucosidase assays in the diagnosis of Gaucher's disease
-
Daniels L.B., and Glew R.H. Beta-glucosidase assays in the diagnosis of Gaucher's disease. Clin Chem 28 (1982) 569-577
-
(1982)
Clin Chem
, vol.28
, pp. 569-577
-
-
Daniels, L.B.1
Glew, R.H.2
-
29
-
-
0035195967
-
Various cells retrovirally transduced with N-acetylgalactosoamine-6-sulfate sulfatase correct Morquio skin fibroblasts in vitro
-
Toietta G., Severini G.M., Traversari C., Tomatsu S., Sukegawa K., Fukuda S., Kondo N., et al. Various cells retrovirally transduced with N-acetylgalactosoamine-6-sulfate sulfatase correct Morquio skin fibroblasts in vitro. Hum Gene Ther 2 (2001) 2007-2016
-
(2001)
Hum Gene Ther
, vol.2
, pp. 2007-2016
-
-
Toietta, G.1
Severini, G.M.2
Traversari, C.3
Tomatsu, S.4
Sukegawa, K.5
Fukuda, S.6
Kondo, N.7
-
30
-
-
0030804213
-
Toward a transgenic mouse model of remyelination
-
Ferraresi S., Lorenzetti I., Nemni R., Kamholz J., Feltri M.L., and Wrabetz L. Toward a transgenic mouse model of remyelination. Mult Scler 3 (1997) 80-83
-
(1997)
Mult Scler
, vol.3
, pp. 80-83
-
-
Ferraresi, S.1
Lorenzetti, I.2
Nemni, R.3
Kamholz, J.4
Feltri, M.L.5
Wrabetz, L.6
-
31
-
-
18144414692
-
Expression and purification of a human, soluble arylsulfatase A for metachromatic leukodystrophy enzyme replacement therapy
-
Martino S., Consiglio A., Cavalieri C., Tiribuzi R., Costanzi E., Severini G.M., et al. Expression and purification of a human, soluble arylsulfatase A for metachromatic leukodystrophy enzyme replacement therapy. J Biotechnol 117 (2005) 243-251
-
(2005)
J Biotechnol
, vol.117
, pp. 243-251
-
-
Martino, S.1
Consiglio, A.2
Cavalieri, C.3
Tiribuzi, R.4
Costanzi, E.5
Severini, G.M.6
-
32
-
-
0028988037
-
Beta-N-acetylhexosaminidases A and S have similar sub-cellular distributions in HL-60 cells
-
Martino S., Emiliani C., Orlacchio A., Hosseini R., and Stirling J.L. Beta-N-acetylhexosaminidases A and S have similar sub-cellular distributions in HL-60 cells. Biochim Biophys Acta 1243 (1995) 489-495
-
(1995)
Biochim Biophys Acta
, vol.1243
, pp. 489-495
-
-
Martino, S.1
Emiliani, C.2
Orlacchio, A.3
Hosseini, R.4
Stirling, J.L.5
-
33
-
-
0017613305
-
Peptide mapping by limited proteolysis in sodium dodecyl sulfate and analysis by gel electrophoresis
-
Cleveland D.W., Fischer S.G., Kirschner M.W., and Laemmli U.K. Peptide mapping by limited proteolysis in sodium dodecyl sulfate and analysis by gel electrophoresis. J Biol Chem 252 (1977) 1102-1106
-
(1977)
J Biol Chem
, vol.252
, pp. 1102-1106
-
-
Cleveland, D.W.1
Fischer, S.G.2
Kirschner, M.W.3
Laemmli, U.K.4
-
34
-
-
0024562391
-
Correlation of the dispersion state of pyrene cerebroside sulfate and its uptake and degradation by cultured cells
-
Viani P., Marchesini S., Cestaro B., and Gatt S. Correlation of the dispersion state of pyrene cerebroside sulfate and its uptake and degradation by cultured cells. Biochim Biophys Acta 1002 (1989) 20-27
-
(1989)
Biochim Biophys Acta
, vol.1002
, pp. 20-27
-
-
Viani, P.1
Marchesini, S.2
Cestaro, B.3
Gatt, S.4
-
35
-
-
0028326747
-
Gene therapy for lysosomal disorders
-
Naffakh N., Bohl D., Salvetti A., Moullier P., Danos O., and Heard J.M. Gene therapy for lysosomal disorders. Nouv Rev Fr Hematol 36 1 (1994) S11-S16
-
(1994)
Nouv Rev Fr Hematol
, vol.36
, Issue.1
-
-
Naffakh, N.1
Bohl, D.2
Salvetti, A.3
Moullier, P.4
Danos, O.5
Heard, J.M.6
-
36
-
-
0031718623
-
Continuous delivery of human and mouse erythropoietin in mice by genetically engineered polymer encapsulated myoblasts
-
Regulier E., Schneider B.L., Deglon N., Beuzard Y., and Aebischer P. Continuous delivery of human and mouse erythropoietin in mice by genetically engineered polymer encapsulated myoblasts. Gene Ther 5 (1998) 1014-1022
-
(1998)
Gene Ther
, vol.5
, pp. 1014-1022
-
-
Regulier, E.1
Schneider, B.L.2
Deglon, N.3
Beuzard, Y.4
Aebischer, P.5
-
37
-
-
0017361716
-
A myogenic cell line with altered serum requirements for differentiation
-
Yaffe D., and Saxel O. A myogenic cell line with altered serum requirements for differentiation. Differentiation 7 (1977) 159-166
-
(1977)
Differentiation
, vol.7
, pp. 159-166
-
-
Yaffe, D.1
Saxel, O.2
-
39
-
-
0029823186
-
Extracellular matrix is required for skeletal muscle differentiation but not myogenin expression
-
Melo F., Carey D.J., and Brandan E. Extracellular matrix is required for skeletal muscle differentiation but not myogenin expression. J.Cell Biochem 62 (1996) 227-239
-
(1996)
J.Cell Biochem
, vol.62
, pp. 227-239
-
-
Melo, F.1
Carey, D.J.2
Brandan, E.3
-
40
-
-
0034869209
-
Retrovirus-mediated gene transfer and galactocerebrosidase uptake into twitcher glial cells results in appropriate localization and phenotype correction
-
Luddi A., Volterrani M., Strazza M., Smorlesi A., Rafi M.A., Datto J., et al. Retrovirus-mediated gene transfer and galactocerebrosidase uptake into twitcher glial cells results in appropriate localization and phenotype correction. Neurobiol Dis 8 (2001) 600-610
-
(2001)
Neurobiol Dis
, vol.8
, pp. 600-610
-
-
Luddi, A.1
Volterrani, M.2
Strazza, M.3
Smorlesi, A.4
Rafi, M.A.5
Datto, J.6
-
41
-
-
0023949553
-
Perivascular microglial cells of the CNS are bone marrow-derived and present antigen in vivo
-
Hickey W.F., and Kimura H. Perivascular microglial cells of the CNS are bone marrow-derived and present antigen in vivo. Science 15 (1988) 290-292
-
(1988)
Science
, vol.15
, pp. 290-292
-
-
Hickey, W.F.1
Kimura, H.2
-
42
-
-
0029146242
-
Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation
-
Shapiro E.G., Lockman L.A., Balthazor M., and Krivit W. Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation. J Inherit Metab Dis 18 (1995) 413-429
-
(1995)
J Inherit Metab Dis
, vol.18
, pp. 413-429
-
-
Shapiro, E.G.1
Lockman, L.A.2
Balthazor, M.3
Krivit, W.4
-
43
-
-
0037295890
-
Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines
-
Peters C., and Steward C.G. Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant 31 (2003) 229-239
-
(2003)
Bone Marrow Transplant
, vol.31
, pp. 229-239
-
-
Peters, C.1
Steward, C.G.2
-
44
-
-
0031019586
-
Adenovirus-mediated gene transfer and expression of human-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice
-
Ohashi T., Watabe K., Uehara K., Sly W.S., Vogler C., and Eto Y. Adenovirus-mediated gene transfer and expression of human-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice. Proc Natl Acad Sci USA 94 (1997) 1287-1292
-
(1997)
Proc Natl Acad Sci USA
, vol.94
, pp. 1287-1292
-
-
Ohashi, T.1
Watabe, K.2
Uehara, K.3
Sly, W.S.4
Vogler, C.5
Eto, Y.6
-
45
-
-
0033642895
-
Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors
-
Bosch A., Perret E., Desmaris N., and Heard J.M. Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors. Mol Ther 1 (2000) 63-71
-
(2000)
Mol Ther
, vol.1
, pp. 63-71
-
-
Bosch, A.1
Perret, E.2
Desmaris, N.3
Heard, J.M.4
-
46
-
-
0031031008
-
GDNF reduces drug-induced rotational behavior after medial forebrain bundle transection by a mechanism not involving striatal dopamine
-
Tseng J.L., Baetge E.E., Zurn A.D., and Aebischer P. GDNF reduces drug-induced rotational behavior after medial forebrain bundle transection by a mechanism not involving striatal dopamine. J Neurosci 17 (1997) 325-333
-
(1997)
J Neurosci
, vol.17
, pp. 325-333
-
-
Tseng, J.L.1
Baetge, E.E.2
Zurn, A.D.3
Aebischer, P.4
-
47
-
-
0033917280
-
Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease
-
Leone P., Janson C.G., Bilaniuk L., Wang Z., Sorgi F., Huang L., et al. Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease. Ann Neurol 48 (2000) 27-38
-
(2000)
Ann Neurol
, vol.48
, pp. 27-38
-
-
Leone, P.1
Janson, C.G.2
Bilaniuk, L.3
Wang, Z.4
Sorgi, F.5
Huang, L.6
-
48
-
-
0029761786
-
Implants of encapsulated human CNTF-producing fibroblasts prevent behavioral deficits and striatal degeneration in a rodent model of Huntington's disease
-
Emerich D.F., Lindner M.D., Winn S.R., Chen E.Y., Frydel B.R., and Kordower J.H. Implants of encapsulated human CNTF-producing fibroblasts prevent behavioral deficits and striatal degeneration in a rodent model of Huntington's disease. J Neurosci 16 (1996) 5168-51681
-
(1996)
J Neurosci
, vol.16
, pp. 5168-51681
-
-
Emerich, D.F.1
Lindner, M.D.2
Winn, S.R.3
Chen, E.Y.4
Frydel, B.R.5
Kordower, J.H.6
-
49
-
-
0030906578
-
Protective effect of encapsulated cells producing neurotrophic factor CNTF in a monkey model of Huntington's disease
-
Emerich D.F., Winn S.R., Hantraye P.M., Peschanski M., Chen E.Y., Chu Y., et al. Protective effect of encapsulated cells producing neurotrophic factor CNTF in a monkey model of Huntington's disease. Nature 386 (1997) 395-399
-
(1997)
Nature
, vol.386
, pp. 395-399
-
-
Emerich, D.F.1
Winn, S.R.2
Hantraye, P.M.3
Peschanski, M.4
Chen, E.Y.5
Chu, Y.6
-
50
-
-
0032928402
-
Delivery of recombinant gene products to the central nervous system with nonautologous cells in alginate microcapsules
-
Ross C.J., Ralph M., and Chang P.L. Delivery of recombinant gene products to the central nervous system with nonautologous cells in alginate microcapsules. Hum Gene Ther 10 (1999) 49-59
-
(1999)
Hum Gene Ther
, vol.10
, pp. 49-59
-
-
Ross, C.J.1
Ralph, M.2
Chang, P.L.3
-
51
-
-
0038703753
-
Prevention of the initial host immuno-inflammatory response determines the long-term survival of encapsulated myoblasts genetically engineered for erythropoietin delivery
-
Schneider B.L., Schwenter F., Pralong W.F., and Aebischer P. Prevention of the initial host immuno-inflammatory response determines the long-term survival of encapsulated myoblasts genetically engineered for erythropoietin delivery. Mol Ther 7 (2003) 506-514
-
(2003)
Mol Ther
, vol.7
, pp. 506-514
-
-
Schneider, B.L.1
Schwenter, F.2
Pralong, W.F.3
Aebischer, P.4
-
52
-
-
0030690936
-
A gene therapy approach to regulated delivery of erythropoietin as a function of oxygen tension
-
Rinsch C., Regulier E., Deglon N., Dalle B., Beuzard Y., and Aebischer P. A gene therapy approach to regulated delivery of erythropoietin as a function of oxygen tension. Hum Gene Ther 8 (1997) 1881-1889
-
(1997)
Hum Gene Ther
, vol.8
, pp. 1881-1889
-
-
Rinsch, C.1
Regulier, E.2
Deglon, N.3
Dalle, B.4
Beuzard, Y.5
Aebischer, P.6
|